CLINICAL TRIALS PROFILE FOR VEMURAFENIB

All Clinical Trials for Vemurafenib

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00949702 ↗	A Study of Vemurafenib in Previously Treated Patients With Metastatic Melanoma	Completed	Hoffmann-La Roche	Phase 2	2009-09-30	This open-label single arm study will assess the efficacy, safety and tolerability of Vemurafenib in previously treated patients with metastatic melanoma. Patients will receive oral Vemurafenib [RG7204; PLEXXIKON: PLX4032] at a dose of 960 mg b.i.d. continuously until disease progression or withdrawal from study and will be assessed at regular intervals for tumour response and tolerability. Target sample size is <100 patients.
NCT01006980 ↗	A Study of Vemurafenib (RO5185426) in Comparison With Dacarbazine in Previously Untreated Patients With Metastatic Melanoma (BRIM 3)	Completed	Hoffmann-La Roche	Phase 3	2010-01-01	This randomized, open-label study evaluated the efficacy, safety and tolerability of vemurafenib (RO5185426) as compared to dacarbazine in previously untreated patients with metastatic melanoma. Patients were randomized to receive either vemurafenib 960 mg orally twice daily or dacarbazine 1000 mg/m2 intravenously every 3 weeks. Study treatment was continued until disease progression or unacceptable toxicity occurred. The data and safety monitoring board recommended that patients in the dacarbazine group be allowed to cross over to receive vemurafenib, and the protocol was amended accordingly on January 14, 2011, as both overall survival and progression-free survival endpoints had met the prespecified criteria for statistical significance in favor of vemurafenib.
NCT01271803 ↗	A Study of Vemurafenib and GDC-0973 (Cobimetinib) in Participants With BRAFV600E Mutation-Positive Metastatic Melanoma	Completed	Hoffmann-La Roche	Phase 1	2011-02-17	This open-label, dose-escalation study of vemurafenib in combination with cobimetinib will evaluate the safety, tolerability and pharmacokinetics in participants with BRAFV600 mutation-positive metastatic melanoma. Participants with previously untreated, BRAFV600E mutation-positive, locally advanced/unresectable or metastatic melanoma or those who have progressed on vemurafenib monotherapy immediately prior to enrolling in this trial are eligible. Participants will be assigned to different cohorts with escalating oral doses of vemurafenib and cobimetinib. This study consists of 2 stages, Stage 1 (Dose Escalation Stage [DES] and Cohort Expansion Stage [CES]) and the anticipated time on study treatment is until disease progression, unacceptable toxicity or any other discontinuation criterion is met.
NCT01286753 ↗	A Study of Vemurafenib (RO5185426) in Participants With Metastatic or Unresectable Papillary Thyroid Cancer Positive for the BRAF V600 Mutation	Completed	Hoffmann-La Roche	Phase 2	2011-06-01	This open-label, multi-center study will evaluate the safety and efficacy of Vemurafenib (RO5185426) in participants with metastatic or unresectable papillary thyroid cancer (PTC) positive for the BRAF V600 mutation and resistant to radioactive iodine therapy. Participants will receive vemurafenib 960 milligrams (mg) orally twice daily until progressive disease or unacceptable toxicity occurs.
NCT01307397 ↗	A Study of Vemurafenib in Participants With Metastatic Melanoma	Completed	Hoffmann-La Roche	Phase 3	2011-03-01	This multi-center study evaluates the safety and efficacy of vemurafenib in participants with BRAF V600 mutation-positive, surgically incurable, and unresectable Stage IIIC or IV (American Joint Committee on Cancer [AJCC]) metastatic melanoma.
NCT01378975 ↗	A Study of Vemurafenib in Metastatic Melanoma Participants With Brain Metastases	Completed	Hoffmann-La Roche	Phase 2	2011-07-01	This open-label, single-arm, multicenter study will evaluate the efficacy and safety in participants with metastatic melanoma who developed brain metastases. Participants may or may not have received prior systemic treatment for metastatic melanoma [except treatment with v-raf murine sarcoma viral oncogene homolog B (BRAF) or mitogen-activated protein kinase (MEK) inhibitors]. Participants will receive oral doses of 960 mg vemurafenib twice daily until disease progression, unacceptable toxicity or consent withdrawal.
NCT01400451 ↗	Ph I Ipilimumab Vemurafenib Combo in Patients With v-Raf Murine Sarcoma Viral Oncogene Homolog B1 (BRAF)	Terminated	Roche-Genentech	Phase 1	2011-11-01	Treatment of subjects who have metastatic melanoma that expresses an activated mutant form of the BRAF oncogene (V600E) with a combination of the specific BRAF inhibitor, Vemurafenib, and the Cytotoxic T Lymphocyte Antigen 4 (CTLA-4) inhibitor mAb Ipilimumab will be safe and feasible and will show preliminary evidence of anti-tumor efficacy and survival in comparison to historical results following treatment with either agent alone.

Clinical Trial Conditions for Vemurafenib

Condition Name

Condition Name for Vemurafenib
Intervention	Trials
Melanoma	38
Malignant Melanoma	17
Metastatic Melanoma	9
Malignant Melanoma, Neoplasms	6

Condition MeSH

Condition MeSH for Vemurafenib
Intervention	Trials
Melanoma	87
Neoplasms	18
Neoplasm Metastasis	10
Colorectal Neoplasms	10

Clinical Trial Locations for Vemurafenib

Trials by Country

Trials by Country for Vemurafenib
Location	Trials
United States	577
Italy	129
Spain	77
Australia	60
Germany	45

Trials by US State

Trials by US State for Vemurafenib
Location	Trials
California	36
Texas	34
New York	34
Massachusetts	27
Florida	27

Clinical Trial Progress for Vemurafenib

Clinical Trial Phase

Clinical Trial Phase for Vemurafenib
Clinical Trial Phase	Trials
Phase 4	4
Phase 3	9
Phase 2/Phase 3	2

Clinical Trial Status

Clinical Trial Status for Vemurafenib
Clinical Trial Phase	Trials
Completed	51
Recruiting	35
Active, not recruiting	23

Clinical Trial Sponsors for Vemurafenib

Sponsor Name

Sponsor Name for Vemurafenib
Sponsor	Trials
Hoffmann-La Roche	32
Genentech, Inc.	25
National Cancer Institute (NCI)	13

Sponsor Type

Sponsor Type for Vemurafenib
Sponsor	Trials
Other	144
Industry	97
NIH	14

Vemurafenib: Clinical Trials, Market Analysis, and Projections

Introduction to Vemurafenib

Vemurafenib, marketed under the brand name Zelboraf, is a kinase inhibitor specifically designed to treat patients with unresectable or metastatic melanoma who have the BRAF V600E mutation. This mutation is a common genetic alteration in melanoma, leading to uncontrolled cell growth.

Clinical Trials Overview

BRIM3 and BRIM2 Studies

The FDA approval of vemurafenib was based on the results of two significant clinical studies: BRIM3 and BRIM2.

• BRIM3: This phase III study compared vemurafenib (960 mg orally twice daily) with dacarbazine (1000 mg/m² IV every 3 weeks) in 675 patients with previously untreated BRAF V600E mutation-positive, unresectable or metastatic melanoma. The study showed that vemurafenib significantly improved overall survival and progression-free survival compared to dacarbazine. Median overall survival was 13.6 months for vemurafenib versus 10.3 months for dacarbazine, and median progression-free survival was 5.3 months for vemurafenib versus 1.6 months for dacarbazine[1].

• BRIM2: This phase II study enrolled 132 patients with previously treated BRAF V600 mutation-positive metastatic melanoma. The study demonstrated a confirmed best overall response rate of 18% in both cohorts, with a median duration of response of 4.6 to 6.6 months[1].

Ongoing Clinical Trials

Combination Therapy with Rituximab

A phase II open-label single-arm study at Scripps is investigating the efficacy and safety of vemurafenib in combination with rituximab for patients with hairy cell leukemia who have the BRAF V600E mutation. The study uses a lower dose of vemurafenib (240 mg twice daily) compared to the standard dose. Interim results show complete hematologic recovery in all seven patients, with improved blood counts and no significant adverse effects[4].

Combination with Immunotherapy

Clinical trials are also exploring the combination of vemurafenib with other therapies, such as immunotherapies. For example, the IMspire150 trial investigates the combination of atezolizumab (Tecentriq), an anti-PD-L1 immunotherapy, with cobimetinib (Cotellic) and vemurafenib. This triple combination has shown significant improvement in progression-free survival compared to the combination of Cotellic and vemurafenib alone[5].

Market Analysis

Current Market Size and Growth

The global vemurafenib market was valued at USD 200 million in 2023 and is projected to reach USD 318.77 million by 2031, growing at a CAGR of 6% from 2024 to 2031. This growth is driven by several factors, including the rising incidence of melanoma and other malignancies with BRAF V600 mutations, advancements in targeted cancer treatments, and improved diagnostic methods[3].

Key Players

The vemurafenib market is dominated by several key players, including Roche, Fermion, Haohong Biotechnology, LGM Pharma, KeChow Pharma, Hetero, ANQING CHICO, Shanghai Shenyue Biotechnology, and Kaiyu Pharmatech. These companies are involved in the production, distribution, and research of vemurafenib across various regions[2][3].

Market Segments

The market is segmented based on type (tablet, capsule, injection) and application (melanoma, non-small cell lung cancer, Erdheim-Chester disease). Geographically, the market is analyzed across North America, Europe, Asia-Pacific, South America, and the Middle East and Africa[3].

Market Projections

Driving Factors

Several factors are driving the growth of the vemurafenib market:

• Increasing Incidence of Melanoma: The rising rate of melanoma and other cancers with BRAF V600 mutations is a major driver.
• Advancements in Targeted Therapy: Developments in precision medicine and genetic testing enable more accurate identification of patients who can benefit from vemurafenib.
• Combination Therapies: Ongoing clinical trials investigating the potential of vemurafenib in combination with other therapies are expanding its medicinal applications.
• Regulatory Approvals and Reimbursement Policies: Favorable regulatory environments and advantageous reimbursement policies are also contributing to market growth[3].

Challenges and Opportunities

Despite the positive outlook, the market faces challenges such as the high cost of targeted therapies and the potential for resistance to BRAF inhibitors. However, these challenges also present opportunities for further research and development of combination therapies and new treatment strategies.

"Approximately half of melanoma patients harbor a mutation in the BRAF gene, which leads to uncontrolled cell growth. BRAF inhibitors like vemurafenib (Zelboraf) and dabrafenib (Tafinlar) have been designed to inhibit the activity of the mutated BRAF protein, thereby curbing tumor growth."[5]

Conclusion

Vemurafenib has established itself as a crucial treatment option for patients with BRAF V600E mutation-positive melanoma. The drug's efficacy, as demonstrated in clinical trials like BRIM3 and BRIM2, and its ongoing evaluation in combination therapies, underscores its importance in oncology.

Key Takeaways

• Clinical Efficacy: Vemurafenib significantly improves overall survival and progression-free survival in patients with BRAF V600E mutation-positive melanoma.
• Market Growth: The global vemurafenib market is projected to grow at a CAGR of 6% from 2024 to 2031.
• Driving Factors: The market is driven by the increasing incidence of melanoma, advancements in targeted therapy, and favorable regulatory environments.
• Ongoing Research: Clinical trials are exploring the potential of vemurafenib in combination with other therapies, including immunotherapies.

FAQs

What is vemurafenib used for?

Vemurafenib is used for the treatment of patients with unresectable or metastatic melanoma who have the BRAF V600E mutation.

What were the key findings of the BRIM3 and BRIM2 studies?

The BRIM3 study showed that vemurafenib improved overall survival and progression-free survival compared to dacarbazine. The BRIM2 study demonstrated a confirmed best overall response rate of 18% in patients with previously treated BRAF V600 mutation-positive metastatic melanoma.

Who are the key players in the vemurafenib market?

Key players include Roche, Fermion, Haohong Biotechnology, LGM Pharma, KeChow Pharma, Hetero, ANQING CHICO, Shanghai Shenyue Biotechnology, and Kaiyu Pharmatech.

What is the projected market size of vemurafenib by 2031?

The global vemurafenib market is expected to reach USD 318.77 million by 2031, growing at a CAGR of 6% from 2024 to 2031.

What are the driving factors for the growth of the vemurafenib market?

The market is driven by the increasing incidence of melanoma, advancements in targeted therapy, improved diagnostic methods, and favorable regulatory environments.

Sources

1. DermNet: Vemurafenib - DermNet
2. Valuates Reports: Global Vemurafenib Market Research Report 2024
3. Market Research Intellect: Vemurafenib Market Size and Projections
4. Hairy Cell Leukemia Foundation: May 2024 clinical trials Scripps
5. BioSpace: Melanoma Market to Reach USD 8.9 Billion by 2034